A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)

Open to people ages 18 years and up

1. Male or female ≥18 years at the time of signing the informed consent
2. Capable and willing to provide signed informed consent
3. Clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC)
4. Documentation of a pathogenic or likely pathogenic truncating variant in PKP2
5. Anti-AAVrh.74 capsid neutralizing antibody assay ≤1:40
6. History of Implantable Cardioverter-Defibrillator (ICD) implantation ≥6 months prior to enrollment

1. Cardiomyopathy related to a genetic etiology other than PKP2 truncating variant
2. Previous participation in a study of gene transfer or gene editing
3. Severe Right ventricular (RV) dysfunction
4. Left ventricular ejection fraction by echocardiogram ≤50%
5. New York Heart Association (NYHA) Class IV heart failure.